Edgewise Therapeutics Talks Phase III Becker Readout, EDG-7500 HCM Data Due in Q2 at Conference

Executives from Edgewise Therapeutics (NASDAQ:EWTX) highlighted recent clinical updates and upcoming milestones across the ...

Gene Therapy for Duchenne Muscular Dystrophy: Genethon Confirms Two-Year Efficacy in Patients Treated with Its Drug Candidate GNT0004 at Therapeutic Dose in the First Phase of ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...
BioSpace

BridgeBio Builds Case for Early 2027 Launch of Dystrophy Drug

Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular ...
Business Wire

Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) today announced that CIFFREO, a Phase 3 global, multicenter, randomized, double-blind, placebo-controlled study evaluating the investigational ...
Business Wire

Satellos Announces First Participant with Duchenne Muscular Dystrophy Dosed in the Phase 1b Clinical Trial of SAT-3247

TORONTO--(BUSINESS WIRE)--Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a public biotech company developing new small molecule therapeutic approaches to improve the ...

Dyne Therapeutics Announces Initiation of Phase 3 HARMONIA Trial of Z-Basivarsen in Myotonic Dystrophy Type 1 (DM1)

HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...

Solid Biosciences Reports Encouraging Phase 1/2 Duchenne Data

Detailed price information for Solid Biosciences Inc (SLDB-Q) from The Globe and Mail including charting and trades.

Solid Biosciences Provides Interim Positive Clinical Update on Phase 1/2 INSPIRE DUCHENNE Trial

SGT-003 in the INSPIRE DUCHENNE trial to date - - Robust microdystrophin expression with consistent mechanistic evidence of dystrophin associated protein complex (DAPC) restoration and improvements in ...
BioSpace

MDA 2026: Biogen, Sarepta, Edgewise Address Muscle Disorders

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies for spinal muscular atrophy, Duchenne muscular dystrophy and Becker muscular ...
Nasdaq

Capricor Therapeutics and Parent Project Muscular Dystrophy to Host Webinar Highlighting Positive Phase 3 HOPE-3 Topline Results in Duchenne Muscular Dystrophy

SAN DIEGO, Dec. 16, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics, today announced that ...
Seeking Alpha

Cumberland Pharmaceuticals Announces Breakthrough Results from the Phase 2 FIGHT DMD Trial in Duchenne Muscular Dystrophy Heart Disease

NASHVILLE, Tenn., Feb. 4, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today ...
FierceBiotech

PepGen ends all work on Duchenne muscular dystrophy after lead asset fails to raise dystrophin levels

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of PepGen’s lead Duchenne muscular dystrophy (DMD) candidate has resulted in limited ...